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EBMT 2025 | OPTIMIZE trial: lowering PTCy dose to improve outcomes of MMUD transplants

In this video, Steven Devine, MD, National Marrow Donor Program (NMDP), Minneapolis, MN, discusses ongoing clinical research efforts of the NMDP to improve outcomes in recipients of mismatched unrelated donor (MMUD) transplants. Although PTCy helps to prevent graft-versus-host disease (GvHD) in these patients, it is associated with side effects and increased infection risk. Dr Devine highlights the OPTIMIZE study, which aims to assess a lower dose of PTCy to reduce toxicities while maintaining the benefit of GvHD prevention. This interview took place at the 51st Annual Meeting of the EBMT in Florence, Italy.

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Transcript

Our studies have been focused initially on trying to demonstrate that outcomes using a mismatched unrelated donor can be improved if you use post-transplant cyclophosphamide to prevent graft-versus-host disease. But that does come at a cost – PTCy is associated with side effects. There are more toxicities or side effects in the recipients, particularly the older recipients, because of the high doses of cyclophosphamide that are being used...

Our studies have been focused initially on trying to demonstrate that outcomes using a mismatched unrelated donor can be improved if you use post-transplant cyclophosphamide to prevent graft-versus-host disease. But that does come at a cost – PTCy is associated with side effects. There are more toxicities or side effects in the recipients, particularly the older recipients, because of the high doses of cyclophosphamide that are being used. There’s more infections because the PTCy can suppress the immune system. So now our studies are focused on whether we can actually reduce the dose of PTCy to lower the risk of side effects and to lower the risk of infections after transplant and yet still retain the benefit in preventing graft-versus-host disease and we’re conducting a study that’s accruing quite well right now called OPTIMIZE where we’re attempting to do that.

 

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